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Collection and Storage of Umbilical Cord Stem Cells for Treatment of Sickle Cell Disease

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT00012545
Recruitment Status : Recruiting
First Posted : March 12, 2001
Last Update Posted : October 26, 2020
Sponsor:
Information provided by (Responsible Party):
National Institutes of Health Clinical Center (CC) ( National Heart, Lung, and Blood Institute (NHLBI) )

Tracking Information
First Submitted Date March 10, 2001
First Posted Date March 12, 2001
Last Update Posted Date October 26, 2020
Actual Study Start Date November 1, 2001
Primary Completion Date Not Provided
Current Primary Outcome Measures
 (submitted: October 3, 2018)
To procure umbilical cord blood (UCB) from newborns at risk for sickle cell disease, sickle cell trait, and related disorders as well as normal newborns, as controls [ Time Frame: End of study ]
To evaluate the feasibility of performing directed donor umbilical cord blood banking for families at risk for having children with congenital diseases amenable to treatment by autologous gene therapy or allogeneic hematopoietic transplantation; in this protocol, the feasibility will be studied specifically in families with risk for sickle cell anemia and related syndromes.
Original Primary Outcome Measures Not Provided
Change History
Current Secondary Outcome Measures Not Provided
Original Secondary Outcome Measures Not Provided
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
 
Descriptive Information
Brief Title Collection and Storage of Umbilical Cord Stem Cells for Treatment of Sickle Cell Disease
Official Title Collection and Storage of Umbilical Cord Hematopoietic Stem Cells for Sickle Cell Disease Therapy
Brief Summary

This study will determine the best ways to collect, process and store umbilical cord blood from babies with sickle cell disease, sickle cell trait and unaffected babies. Sickle cell disease is an abnormality of the hemoglobin in red blood cells that causes the cells to change shape and clump together, preventing their normal flow in the bloodstream. This impairs blood flow to various organs, and the resulting oxygen deprivation causes organ damage.

Cord blood is rich in stem cells (cells produced in the bone marrow that mature to different types of blood cells), which may prove useful in new sickle cell therapies. However, cord blood from babies with sickle cell trait, sickle cell disease and normal babies may act differently under laboratory conditions, so it is important to learn how best to work with blood from all three groups of babies for future use in possible treatments.

Pregnant women between 18 and 45 years of age who are at risk of having an infant with sickle cell disease and normal volunteers who are pregnant and not at risk for this disease may be eligible for this study. Potential participants will be counseled about donating her infant s blood in order to make an informed choice.

All women who participate in the study will provide a medical history and have blood collected from the umbilical cord and placenta (afterbirth) after the baby s delivery. The blood will be tested for various infectious diseases, processed, frozen and stored for research purposes. In addition, blood from women with babies at risk for sickle cell disease will be tested for the presence of the sickle cell gene, tissue typed, and used for research as follows:

  • Sickle cell disease If cord blood tests show the baby has sickle cell disease, the blood will be frozen for an indefinite period of time for possible use in future treatment of the child. This treatment could include stem cell transplantation or gene therapy, treatments are not currently considered routine for sickle cell disease.
  • Sickle cell trait or normal hemoglobin If cord blood tests show the baby has sickle cell trait or is unaffected, the blood will be processed and stored for up to 3 years, during which time it may possibly be used to treat a currently living or future sibling with sickle cell disease. After 3 years, the participant may agree to either have the blood discarded, given to research or moved to another facility for continued storage at the participant s expense, if there is a storage fee. Alternatively, if there is no anticipated future need for the collected blood, or if it does not meet standards needed for future treatment, it will be used in NIH-approved research studies.

Participants and their family doctor or the baby s pediatrician will be contacted twice a year for information about changes in the baby s health. Participants may also be asked permission to perform new tests developed by researchers.

Detailed Description Umbilical cord blood is a source of hematopoietic stem cells (HSCs) for transplantation or gene therapy. Our goal is to procure umbilical cord blood (UCB) from newborns at risk for sickle cell disease, sickle cell trait, and related disorders as well as normal newborns and store clinical grade cord blood units (CBU) for future use in clinical transplantation or gene therapy. Cord blood units will be collected from an indefinite number of subjects until 50 CBU from newborns with homozygous sickle cell disease have been cryopreserved. Maternal subjects will be identified as being at risk to have an infant with sickle cell disease, will be between the ages of 18 and 45 years, and will meet specified medical history criteria. Mothers will have to deliver at INOVA Fairfax Hospital (collection provider site) and the CB will be collected by NCBP collectors ex utero, will be transported to the NCBP CB facility and will be processed, tested and stored. Frozen CBU will be transferred to the NIH for clinical transplantation or gene therapy studies upon NIH request.
Study Type Observational
Study Design Observational Model: Case-Only
Time Perspective: Cross-Sectional
Target Follow-Up Duration Not Provided
Biospecimen Not Provided
Sampling Method Non-Probability Sample
Study Population This is a pilot feasibility study for which umbilical cord blood samples will be collected and transported to the NIH Clinical Center for our developmental research. Pregnant women whose babies are at risk for sickle cell anemia will be identified and referred to the NIH Research Coordinator for evaluation and entry into the study. The NIH Research Coordinator is experienced in performing patient education and counseling concerning sickle cell disease, and obtaining informed consent.
Condition
  • Sickle Cell Disease
  • Sickle Cell Trait
Intervention Not Provided
Study Groups/Cohorts Healthy Pregnant Volunteers
Pregnant women whose babies are at risk for sickle cell anemia will be identified and referred to the NIH Research Coordinator for evaluation and entry into the study.
Publications *

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status Recruiting
Estimated Enrollment
 (submitted: July 23, 2009)
99999999
Original Enrollment
 (submitted: June 23, 2005)
9999999
Study Completion Date Not Provided
Primary Completion Date Not Provided
Eligibility Criteria
  • INCLUSION CRITERIA:

Pregnant women who are at risk of having an infant with sickle cell anemia related diseases.

The types of sickle cell disease include the following:

  • Hemoglobin S <=0 thalassemia
  • Hemoglobin S <=+ thalassemia
  • Hemoglobin SC
  • Hemoglobin SD
  • Hemoglobin SE
  • Hemoglobin SS

    • Maternal subjects must be between 18 and 45 years old.
    • Maternal subjects must be able to provide informed consent.
    • Maternal subjects deliver at INOVA Fairfax Hospital (insurance approval)
    • Maternal subjects enrolled on other studies are not excluded, unless the other study may interfere with the present one.
    • Maternal subjects in active labor will be enrolled at the discretion of her physician. (Note: in rare cases that a mother is identified as a potential candidate for the study during labor, it is at the discretion of her physician whether she could be in a condition to discuss about the study and provide informed consent (then IFH has to obtain the Informed Consent). After labor and delivery, the study will be discussed again with the mother.)

EXCLUSION CRITERIA:

  • The maternal subject will not be eligible for study if she is known to be positive for one or more of the following diseases transmissible by blood: HIV, hepatitis B, hepatitis C, or HTLV or ZIKV.
  • Maternal subject will not be eligible for the study if she has active syphilis, toxoplasmosis, malaria or babesia during pregnancy.
  • Maternal subject will not be eligible for the study if the pregnancy resulted from egg donation or sperm donation.
  • Maternal subject is unable to give informed consent.
  • Maternal subject is known to have a fetus with a significant congenital anomaly, either not compatible with life, or requiring immediate surgical intervention or care in the Neonatal Intensive Care Unit or.
  • Maternal subject delivers at another facility, not INOVA Fairfax Hospital.
  • Maternal subject has cancer or received treatment for cancer during pregnancy
  • Maternal subject may be excluded at the time of delivery if the attending physician or collection staff deems that the cord blood collection is inadvisable, due to unanticipated obstetrical complications.
Sex/Gender
Sexes Eligible for Study: Female
Ages up to 45 Years   (Child, Adult)
Accepts Healthy Volunteers Yes
Contacts
Contact: Wynona Coles (301) 402-2104 wcoles@nhlbi.nih.gov
Contact: John F Tisdale, M.D. (301) 402-6497 johntis@mail.nih.gov
Listed Location Countries United States
Removed Location Countries  
 
Administrative Information
NCT Number NCT00012545
Other Study ID Numbers 010122
01-H-0122
Has Data Monitoring Committee Not Provided
U.S. FDA-regulated Product Not Provided
IPD Sharing Statement Not Provided
Responsible Party National Institutes of Health Clinical Center (CC) ( National Heart, Lung, and Blood Institute (NHLBI) )
Study Sponsor National Heart, Lung, and Blood Institute (NHLBI)
Collaborators Not Provided
Investigators
Principal Investigator: John F Tisdale, M.D. National Heart, Lung, and Blood Institute (NHLBI)
PRS Account National Institutes of Health Clinical Center (CC)
Verification Date October 9, 2020